Pulmonary fibrosis model work
A 2023 in vitro study reported that elamipretide attenuated idiopathic pulmonary fibrosis by inhibiting the Nrf2 dependent NLRP3 inflammasome in macrophages. Reported in the cited paper.
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SS-31, known as elamipretide, is a mitochondria targeted tetrapeptide that binds cardiolipin in the inner mitochondrial membrane. It received FDA accelerated approval in September 2025 as Forzinity for Barth syndrome, the first approved mitochondria targeted therapeutic.
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Overview
SS-31, known as elamipretide, is a mitochondria targeted tetrapeptide that binds cardiolipin in the inner mitochondrial membrane. It received FDA accelerated approval in September 2025 as Forzinity for Barth syndrome, the first approved mitochondria targeted therapeutic.
What the research reports
Outcomes described across the published SS-31 (Elamipretide) literature and ongoing study. Read each as research context, not a promised result.
Cardiolipin stabilisation
Binds cardiolipin in the inner mitochondrial membrane, restoring the structural integrity that efficient electron transport chain function depends upon.
Approved Barth syndrome use
Granted FDA accelerated approval in September 2025 to improve muscle strength in Barth syndrome patients weighing at least 30 kilograms. Reported in research settings.
Muscle strength trial data
The approval rested on knee extensor strength improving by more than 45 percent, a change that correlated with six minute walk results. Reported in research settings.
Oxidative stress reduction
Preclinical work reports reduced reactive oxygen species production, which follows from improved electron transport chain efficiency. Reported in research settings.
Ongoing indication research
Studies continue in dry age related macular degeneration and primary mitochondrial myopathy, neither of which is an approved use. Reported in research settings.
Dosage and protocol
Clinical trials studied 4 to 40 mg once daily by subcutaneous injection across 4 to 12 week protocols. The approved Barth syndrome product has its own labelled dosing schedule.
Clinical evidence
The published record, labeled by strength so you can see what is settled and what is still emerging.
A 2023 in vitro study reported that elamipretide attenuated idiopathic pulmonary fibrosis by inhibiting the Nrf2 dependent NLRP3 inflammasome in macrophages. Reported in the cited paper.
SourceA 2024 in vitro study used elamipretide to counter doxycycline induced mitochondrial dysfunction in aortic smooth muscle cells. As reported in the cited publication.
SourceThe approval drew on the TAZPOWER extension and a phase 3 natural history control study, with an FDA committee voting 10 to 6 that it is effective. Reported in the cited paper.
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